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filingDate 2019-12-20-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationDate 2022-03-07-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber JP-2022517174-A
titleOfInvention Composition for DRG-specific reduction of introduced gene expression
abstract Recombinant AAV (rAAV) comprising an AAV capsid and a vector genome packaged therein is provided herein in which the vector genome is a 5'reverse end repeat (ITR) of AAV in a target cell. Includes an engineered nucleic acid sequence encoding a gene product for expression, and a miRNA target sequence that selectively suppresses expression in posterior root ganglion (DRG) cells. Also provided are pharmaceutical compositions containing rAAV described herein in a pharmaceutical buffer, as well as methods of treating human subjects with CNS-targeted gene therapy while selectively preventing expression in DRG cells. [Selection diagram] FIG. 1A
priorityDate 2018-12-21-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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